Cerebral palsy is a neuromuscular disorder caused mostly by injury or abnormal development in the brain, mostly before birth. John Drambel, who turns 10 in March, will travel to the University of Texas at Houston Tuesday, where the $1.7 million blind study will take place starting Jan. 15. It is for patients ages 2 to 10 and will last one to two years.
John is a quadriplegic and requires round-the-clock care. He cannot dress or use the bathroom himself, and must communicate through a device on his wheelchair. Ed Drambel, John’s father, said there are other studies being conducted at Duke University in Durham, N.C., and at the Medical College of Georgia in Augusta, both using bank umbilical cord blood. But this is the first study of its kind using the patients’ own cells from their hip bones.
“Only 2 percent of the population banks their cord blood,” Ed Drambel said. “This is a therapy that can be available for all with cerebral palsy.”
In recent years there was one study each in South Korea and Mexico, where patients saw a 30 percent improvement rate in their health, he said. Drambel and friend Lizette Dunay co-founded Let’s Cure CP, a national, Atlanta-based nonprofit dedicated to fundraising for research and treatment of the disorder. She and her husband Dave even took their son Alex to Mexico in March 2011 to participate in that study.
The Houston study will include 30 patients, half of which will be treated with cord blood stem cells and the other half with bone marrow stem cells. A third of the patients in each group will receive a placebo, and those who get it can opt to re-enroll in the study to receive the actual stem cells, Ed Drambel said.
“The reason why this is such an important trial is it will compare the effectiveness of cord blood-derived stem cells versus the bone marrow-derived stem cells,” he said. “It’s not just for John but for all the people with cerebral palsy. We’re hoping to see there is improvement in patients that are doing this for a long-term basis.
“There are studies around the world that have shown this. We just need to do it in the United States and build on that. We don’t know what the stem cell supply is or the delivery site, but we have to start somewhere.”
He said Let’s Cure CP has donated $100,000 to the study and is also looking at funding other studies in the future, including possibly one to determine if there is a genetic link to cerebral palsy.